Planning Phase for a Phase IIa Study of Nilotinib in People with Parkinson's Disease

Research Grant, 2016

Study Rationale:

Nilotinib, a drug approved for chronic myelogenous leukemia, inhibits the protein c-Abl. Higher brain levels of c-Abl are associated with Parkinson's disease (PD), and laboratory studies have linked c-Abl to cellular pathways believed to underlie PD. Nilotinib has also shown protective effects in PD preclinical models. Additionally, a small Phase I trial reported nilotinib was well-tolerated and led to improvements in motor skills and cognitive function in participants with advanced Parkinson's disease and Lewy body dementia. However, as the trial was open label (no placebo control group), additional research is needed to understand the potential clinical benefit of nilotinib for PD.

Hypothesis:

The planned trial will test the hypothesis of whether nilotinib is safe and tolerable and will explore potential clinical benefits for people with PD.